Cystinosis is a rare, inherited, metabolic disease that is characterized by the abnormal accumulation of the amino acid cystine in each cell. The build-up of cystine in the cells slowly and eventually destroys all major organs of the body including the kidneys, liver, eyes, muscles, bone marrow, thyroid and brain. Although medication is available to control some of the symptoms of this insidious disease, Cystinosis remains incurable.
Thursday, January 20, 2011
Finally, an update
We continue to make amazing progress on the research front. We have just completed stage 3 clinical trials on a 12 hour dose and will be completing stage 4 in 2011. Our vision of a better quality of life for these patients is right around the corner and we owe all of you a huge debt of gratitude.
In addition, our gene therapy trials continue to show outstanding progress and we are looking to administer human testing within the year!
Our yearly event is coming up February 12th at Charlotte Country Club, we hope to see you there!